TOKYO, Mar 18 (Pulse News Wire) – RIBOMIC Inc. (4591.T) submitted a clinical trial application to the Pharmaceuticals and Medical Devices Agency (PMDA) for its umedaptanib pegol treatment targeting pediatric patients aged 2 to 14 with achondroplasia.
Following PMDA's 14-day review period, the trial will commence. The Phase III study aims to verify the efficacy, safety, and pharmacokinetics of umedaptanib pegol through a non-blinded, multicenter design involving weekly subcutaneous injections of 1 mg/kg over 78 weeks. Key evaluation metrics include changes in annual height velocity (AHV) post-treatment compared to baseline observations. The trial targets 16 cases.
Umedaptanib pegol, designated as RBM-007, is an aptamer designed to inhibit fibroblast growth factor 2 (FGF2) function, offering a potential fundamental therapy for achondroplasia. It has received orphan drug designation from the Ministry of Health, Labour and Welfare. Achondroplasia affects approximately one in every 25,000 newborns, characterized by genetic mutations in FGFR3 leading to excessive FGF signaling and impaired skeletal development. Effective treatments are highly sought after due to the condition’s rarity and severity.
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